A Localized Materials-Based Strategy to Non-Virally Deliver Chondroitinase ABC mRNA Improves Hindlimb Function in a Rat Spinal Cord Injury Model

Adv Healthc Mater. 2022 Oct;11(19):e2200206. doi: 10.1002/adhm.202200206. Epub 2022 Aug 25.

Abstract

Spinal cord injury often results in devastating consequences for those afflicted, with very few therapeutic options. A central element of spinal cord injuries is astrogliosis, which forms a glial scar that inhibits neuronal regeneration post-injury. Chondroitinase ABC (ChABC) is an enzyme capable of degrading chondroitin sulfate proteoglycan (CSPG), the predominant extracellular matrix component of the glial scar. However, poor protein stability remains a challenge in its therapeutic use. Messenger RNA (mRNA) delivery is an emerging gene therapy technology for in vivo production of difficult-to-produce therapeutic proteins. Here, mineral-coated microparticles as an efficient, non-viral mRNA delivery vehicles to produce exogenous ChABC in situ within a spinal cord lesion are used. ChABC production reduces the deposition of CSPGs in an in vitro model of astrogliosis, and direct injection of these microparticles within a glial scar forces local overexpression of ChABC and improves recovery of motor function seven weeks post-injury.

Keywords: biomaterials; messenger RNA delivery; non-viral gene therapy; regenerative medicines; spinal cord injury.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Chondroitin ABC Lyase* / metabolism
  • Chondroitin ABC Lyase* / pharmacology
  • Chondroitin ABC Lyase* / therapeutic use
  • Chondroitin Sulfate Proteoglycans / metabolism
  • Chondroitin Sulfate Proteoglycans / therapeutic use
  • Gliosis / drug therapy
  • Hindlimb / pathology
  • Nerve Regeneration
  • RNA, Messenger / genetics
  • Rats
  • Rats, Sprague-Dawley
  • Spinal Cord / pathology
  • Spinal Cord Injuries* / drug therapy
  • Spinal Cord Injuries* / pathology

Substances

  • Chondroitin Sulfate Proteoglycans
  • RNA, Messenger
  • Chondroitin ABC Lyase